Rocket Pharma's Gene Therapy Future Clouded By Regulatory Uncertainty - Rocket Pharmaceuticals (NASDAQ:RCKT)

Rocket Pharma's Gene Therapy Future Clouded By Regulatory Uncertainty - Rocket Pharmaceuticals (NASDAQ:RCKT)

Rocket Pharmaceuticals faces trial delays, multiple downgrades, and reduced success expectations following RP-A501 safety concerns.

Death in Rocket Pharma Gene Therapy Study Puts Spotlight on Trial Change Intended to Improve Safety

Rocket Pharmaceuticals adjusted the clinical trial design to include use of approved drug to avert an excessive immune response to its Danon disease gene therapy. The patient who died was one of two who received this drug under the revised protocol. The post Death in Rocket Pharma Gene Therapy Study Puts Spotlight on Trial Change Intended to Improve Safety appeared first on MedCity News.

Participant in Rocket Pharmaceuticals gene therapy study dies

Rocket Pharmaceuticals said Tuesday that a patient had died in a trial studying its experimental gene therapy for a rare heart condition called Danon disease.   Rocket did not disclose the patient’s age, although the Phase 2 study aimed to enroll boys and young men. The company said it was still investigating the cause of the death. It suspects a drug given to suppress patients’ immune systems before receiving the gene therapy may have played a …

Patient dies in trial of Rocket gene therapy for heart condition

The FDA has placed a clinical hold on the study while Rocket works with trial monitors and experts to investigate the cause of the patient’s death.

Rocket Pharma stock falls on FDA clinical hold (RCKT:NASDAQ)

Rocket Pharmaceuticals (RCKT) stock faces a 62% stock drop after FDA's hold on the company's Phase 2 gene therapy trial for Danon disease. Read more here.

Danon Disease Patient Dies in Rocket Gene Therapy Trial

Rocket Pharmaceuticals acknowledged the death of a patient in a pivotal trial assessing its Danon disease gene therapy candidate RP-A501, a study that the FDA has placed on clinical hold. The patient, whose age was not disclosed, suffered an unexpected serious adverse event (SAE) that involved clinical complications related to a capillary leak syndrome (CLS), following dosing with RP-A501 in early May, Rocket’s CEO Gaurav Shah, MD, told analysts…